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Lambert-Eaton myasthenic syndrome (LEMS), also known as myasthenic syndrome, is a rare, often tumor-associated autoimmune disease involving the presynaptic membrane of the neuromuscular junction that was first described in 1956. Typical clinical symptoms include progressive truncal muscle weakness, ptosis, as well as diplopia, dysarthria, and dysphagia. The disease is caused by pathogenic autoantibodies that target and inhibit the P/Q-type pressure-gated calcium channels (VGCCs) presynaptic to nerve terminals, which is different from the postsynaptic targets of pathogenic autoantibodies that cause myasthenia gravis (MG).
Neville E. Sanjana's team at New York University published a research paper titled "Transcriptome-scale RNA-targeting CRISPR screens reveal essential lncRNAs in human cells" in the international academic journal Cell. The study developed a transcriptome-scale CRISPR screening technology based on CRISPR-Cas13 targeting RNA, and used this technology to screen and identify 778 essential lncRNAs in 5 human cells from different tissues, indicating that many lncRNAs are not junk, but play an essential and important role in human cancer and development.
In October 2024, Victor Ambros and Gary Ruvkun were awarded the Nobel Prize in Physiology or Medicine for their discovery of the central role of microRNA (miRNA) in gene expression. This discovery in 1993 revealed that miRNA regulates gene expression by binding to target mRNA and inhibiting its translation. Since then, the important role of miRNA in multiple gene expression pathways such as cell differentiation, proliferation and survival has gradually been recognized. However, although the biological functions of miRNA have been well studied, the road to applying miRNA in clinical treatment is still long and challenging.
The Proceedings of the National Academy of Sciences (PNAS) published online a research paper titled "AMBRA1 controls the translation of immune-specific genes in T lymphocytes" by the research group of Yikun Yao from the Shanghai Institute of Nutrition and Health, Chinese Academy of Sciences. This study screened and identified the key regulatory protein AMBRA1 in the FAS-mediated T cell death process, revealing a new mechanism by which AMBRA1 controls TCR signaling, T cell cycle and T cell death at the translation level.
CRISPR-Cas9 has long been likened to a pair of genetic scissors because of its ability to elegantly and precisely snip any desired DNA fragment. But it turns out that the CRISPR system has more than just one strategy in its toolbox. CRISPR is a mechanism originally discovered in bacteria, and it has been operating for centuries as an adaptive immune system. Certain single-celled organisms naturally use CRISPR to protect themselves from viruses (called bacteriophages) and other foreign genetic fragments.
Recently, Nature reported on the headlines of its official website a special case of a virologist who used a virus she cultivated in the laboratory to treat her own cancer.
Recently, researchers from Peking University in China published a research paper titled "Transfer of mitochondrial DNA into the nuclear genome during induced DNA breaks" in the journal Nature Communications. Hu Jiazhi's team used PEM-seq, a high-throughput sequencing method they had previously developed for systematic analysis of gene editing products, and found that mitochondrial DNA fragments may be inserted into the targeted site during nuclear genome editing. At the same time, targeted editing of mitochondrial DNA can also cause mitochondrial DNA instability, leading to its insertion into the nuclear genome.
Recently, researchers published a research paper titled "Engineered IscB-ωRNA system with improved base editing efficiency for disease correction via single AAV delivery in mice" in the journal Cell Reports. The study successfully engineered the IscB-ωRNA system, a transposon-related CRISPR ancestral system. The gene knockout and base editing efficiency of IscB-ωRNA were improved, and the feasibility of the optimized IscB-ωRNA system for gene editing therapy was verified in a mouse metabolic disease model. The optimized micro-IscB-ωRNA system can be delivered via a single adeno-associated virus (AAV) vector, and has great application potential in gene editing therapy.
Chronic viral hepatitis is a long-term inflammation of the liver, usually caused by hepatitis B (HBV), C (HCV) or D (HDV) viruses. Although these viruses replicate differently, they all rely on the endoplasmic reticulum-Golgi pathway for replication. In this process, a gene called TM6SF2 plays a key role. It is estimated that 450,000 people in Australia suffer from chronic viral hepatitis, and about 1,000 people die each year.
The Liu Ruqian team at the Broad Institute published a research paper titled "Directed evolution of engineered virus-like particles with improved production and transduction efficiencies" in the journal Nature Biotechnology. The study used directed evolution to develop the fifth generation of engineered virus-like particles (v5 eVLP), which has higher production efficiency and delivery efficiency.
